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FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen

Big news in exon skipping therapy today! BioMarin announced this morning that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.

We are such a fortunate community to have multiple companies working on multiple therapies to treat people with Duchenne. As always, PPMD will do everything we can to ensure that these therapies are reviewed as rapidly as possible. We are grateful to BioMarin and Sarepta for their commitment to Duchenne.

BioMarin Announces FDA Accepts Drisapersen NDA for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

SAN RAFAEL, Calif., June 29, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.

In the FDA's filing communication, the Agency informed the company that it is currently planning to hold an advisory committee meeting to discuss the application. No date has been set for this meeting. Drisapersen previously has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designations by the FDA. The U.S. filing is based on three randomized placebo-controlled trials and two long-term open-label studies of more than 300 patients in which some boys have been treated for more than three years.

"We are dedicated to bringing a meaningful therapy specifically for patients with a particular form of Duchenne to patients all over the world. We are thrilled that BioMarin has reached this important step in the United States, which comes on the heels of the recent validation of our European filing for drisapersen," said Camilla V. Simpson, Global Head of Regulatory Affairs, Pharmacovigilance at BioMarin. "Obtaining Priority Review status is validation of BioMarin's commitment to urgently move treatment beyond supportive care and to address the underlying cause of the disease. We are thankful to the boys and their families who participated in our clinical trials, which have allowed us to achieve this important milestone for Duchenne patients."

Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of DMD amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to DMD patients for whom skipping of exon 51 restores the proper dystrophin reading frame, corresponding to approximately 13% of DMD patients. In the U.S., it is estimated there are approximately 2,000 patients who would be candidates for drisapersen.

"Since PPMD's founding more than two decades ago, we have been focused on improving the treatment, quality of life and long-term outlook for boys with Duchenne muscular dystrophy," said Pat Furlong, President and Founder of Parent Project Muscular Dystrophy. "The completion of this regulatory milestone brings the community one step closer to what could be the first specific drug therapy to treat Duchenne in the United States. We are hopeful that this is the beginning of a new era of many medical advancements that will change the course of this devastating disease."

View full press release from BioMarin.

Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 29, 2015-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the completion of the rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for eteplirsen on June 26, 2015. Eteplirsen, the Company’s lead drug candidate, targets the underlying cause of Duchenne muscular dystrophy and is designed to enable the production of a functional internally truncated dystrophin protein in patients with mutations amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation targeted by Eteplirsen/exon 51 skipping.

“The completion of our NDA submission for eteplirsen represents the culmination of the efforts of our employees, investigators, clinical trial sites, and most importantly the patients and families of the Duchenne community,” said Edward M. Kaye, interim chief executive officer and chief medical officer. “We look forward to working with the FDA during the regulatory process in pursuit of our goal of bringing eteplirsen to patients amenable to exon 51 skipping, while maintaining our organizational focus on advancing our PMO technology to target other DMD subpopulations amenable to exon-skipping as quickly as possible.”

The NDA submission includes a request for Priority Review. Previously, eteplirsen has been granted Orphan and Fast Track status by the FDA.

The rolling submission of the NDA began on May 20, 2015, after the completion of a pre-NDA meeting with the FDA held on May 19, 2015.

View full press release from Sarepta.

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